Two applications (10%)– lonafarnib (for Hutchinson-Gilford Progeria syndrome [HGPS] and processing deficient progeroid laminopathies) [26] and cerliponase alfa (for neuronal ceroid lipofuscinosis type 2 [CLN2]) [42]– used registry-based natural history data/cohorts for comparison. The gene discussed is TPP1; the disease is Hutchinson-Gilford progeria syndrome.